EP2906696A4 - Procédés pour moduler l'expression de c90rf72 - Google Patents
Procédés pour moduler l'expression de c90rf72Info
- Publication number
- EP2906696A4 EP2906696A4 EP13846313.8A EP13846313A EP2906696A4 EP 2906696 A4 EP2906696 A4 EP 2906696A4 EP 13846313 A EP13846313 A EP 13846313A EP 2906696 A4 EP2906696 A4 EP 2906696A4
- Authority
- EP
- European Patent Office
- Prior art keywords
- c90rf72
- modulating
- expression
- methods
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
- C12N2310/3181—Peptide nucleic acid, PNA
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Veterinary Medicine (AREA)
- Psychiatry (AREA)
- Public Health (AREA)
- Hospice & Palliative Care (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Steroid Compounds (AREA)
- Liquid Crystal Substances (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201261714139P | 2012-10-15 | 2012-10-15 | |
PCT/US2013/065067 WO2014062686A1 (fr) | 2012-10-15 | 2013-10-15 | Procédés pour moduler l'expression de c90rf72 |
Publications (4)
Publication Number | Publication Date |
---|---|
EP2906696A1 EP2906696A1 (fr) | 2015-08-19 |
EP2906696A4 true EP2906696A4 (fr) | 2016-06-08 |
EP2906696B1 EP2906696B1 (fr) | 2019-10-09 |
EP2906696B2 EP2906696B2 (fr) | 2022-12-14 |
Family
ID=50488699
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EP13846313.8A Active EP2906696B2 (fr) | 2012-10-15 | 2013-10-15 | Procédés pour moduler l'expression de c90rf72 |
Country Status (7)
Country | Link |
---|---|
US (1) | US9963699B2 (fr) |
EP (1) | EP2906696B2 (fr) |
DK (1) | DK2906696T4 (fr) |
ES (1) | ES2762326T5 (fr) |
FI (1) | FI2906696T4 (fr) |
PT (1) | PT2906696T (fr) |
WO (1) | WO2014062686A1 (fr) |
Families Citing this family (41)
Publication number | Priority date | Publication date | Assignee | Title |
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EP2370451B1 (fr) | 2008-12-02 | 2016-11-16 | Wave Life Sciences Japan, Inc. | Procédé pour la synthèse d'acides nucléiques modifiés au niveau de l'atome de phosphore |
IN2012DN00720A (fr) | 2009-07-06 | 2015-06-19 | Ontorii Inc | |
WO2012039448A1 (fr) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | Groupe auxiliaire asymétrique |
RU2014105311A (ru) | 2011-07-19 | 2015-08-27 | Уэйв Лайф Сайенсес Пте. Лтд. | Способы синтеза функционализованных нуклеиновых кислот |
EP2872485B1 (fr) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Groupe auxiliaire asymétrique |
CN104968783B (zh) | 2012-10-15 | 2019-12-10 | Ionis制药公司 | 用于调节c9orf72表达的组合物 |
WO2014062736A1 (fr) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Procédés permettant de surveiller l'expression de c9orf72 |
US9963699B2 (en) | 2012-10-15 | 2018-05-08 | Ionis Pharmaceuticals, Inc. | Methods for modulating C9ORF72 expression |
MX2016004651A (es) * | 2013-10-11 | 2016-08-05 | Ionis Pharmaceuticals Inc | Composiciones para modular la expresion de c9orf72. |
US11162096B2 (en) | 2013-10-14 | 2021-11-02 | Ionis Pharmaceuticals, Inc | Methods for modulating expression of C9ORF72 antisense transcript |
KR20240164968A (ko) | 2014-01-16 | 2024-11-21 | 웨이브 라이프 사이언시스 리미티드 | 키랄 디자인 |
WO2016024205A1 (fr) * | 2014-08-15 | 2016-02-18 | Pfizer Inc. | Oligomères ciblant l'expansion d'une répétition hexanucléotidique dans le gène c9orf72 humain |
WO2016112132A1 (fr) | 2015-01-06 | 2016-07-14 | Ionis Pharmaceuticals, Inc. | Compositions destinées à moduler l'expression du transcrit antisens c9orf72 |
HUE049233T2 (hu) * | 2015-04-16 | 2020-09-28 | Ionis Pharmaceuticals Inc | C9ORF72-expresszió módosítására szolgáló készítmények |
WO2016167780A1 (fr) | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions destinées à moduler l'expression du transcrit antisens c9orf72 |
ES2784360T3 (es) | 2015-05-29 | 2020-09-24 | Regeneron Pharma | Animales no humanos que tienen una interrupción en un locus C9ORF72 |
EP4285912A3 (fr) | 2015-09-25 | 2024-07-10 | Ionis Pharmaceuticals, Inc. | Compositions et méthodes de modulation de l'expression de l'ataxine 3 |
US10760076B2 (en) | 2015-10-05 | 2020-09-01 | Proqr Therapeutics Ii B.V. | Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion |
WO2017079291A1 (fr) * | 2015-11-02 | 2017-05-11 | Ionis Pharmaceuticals, Inc. | Composés et méthodes de modulation de c90rf72 |
EP3443094B1 (fr) * | 2016-04-13 | 2022-10-19 | Ionis Pharmaceuticals, Inc. | Procédés de réduction de l'expression de c9orf72 |
RU2760877C2 (ru) | 2016-09-30 | 2021-12-01 | Регенерон Фармасьютикалс, Инк. | Животные, отличные от человека, характеризующиеся экспансией гексануклеотидных повторов в локусе c9orf72 |
JOP20190104A1 (ar) | 2016-11-10 | 2019-05-07 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن atxn3 |
US20180193270A1 (en) | 2016-11-29 | 2018-07-12 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
GB2579747B8 (en) | 2017-08-04 | 2022-10-05 | Skyhawk Therapeutics Inc | Methods and compositions for modulating splicing |
TW201920672A (zh) * | 2017-08-08 | 2019-06-01 | 新加坡商波濤生命科學有限公司 | 寡核苷酸組合物及其使用方法 |
CA3079727A1 (fr) * | 2017-10-24 | 2019-05-02 | Sangamo Therapeutics, Inc. | Methodes et compositions pour le traitement de maladies rares |
US10905707B2 (en) | 2017-11-10 | 2021-02-02 | University Of Massachusetts | Compositions and methods for the treatment of expanded repeat-associated disorders |
CR20200604A (es) | 2018-05-09 | 2021-02-09 | Ionis Pharmaceuticals Inc | Compuestos y métodos para reducir de la expresión de atxn3 |
CN113365640A (zh) | 2018-12-06 | 2021-09-07 | 比奥根Ma公司 | 引导肌萎缩侧索硬化症的治疗性干预的神经丝蛋白 |
EP3732291A1 (fr) | 2018-12-20 | 2020-11-04 | Regeneron Pharmaceuticals, Inc. | Expansion de répétition à médiation par nucléase |
WO2020163405A1 (fr) | 2019-02-05 | 2020-08-13 | Skyhawk Therapeutics, Inc. | Procédés et compositions pour moduler l'épissage |
CN113677344A (zh) | 2019-02-06 | 2021-11-19 | 斯基霍克疗法公司 | 用于调节剪接的方法和组合物 |
KR20210124374A (ko) | 2019-02-08 | 2021-10-14 | 듀포인트 테라퓨틱스, 인크. | 화합물의 응축물-연관된 특징을 특징규명하는 방법 및 그의 용도 |
JP2022532169A (ja) * | 2019-05-09 | 2022-07-13 | ウェイブ ライフ サイエンシズ リミテッド | オリゴヌクレオチド組成物及びその使用方法 |
EP4031876A1 (fr) | 2019-09-18 | 2022-07-27 | Dewpoint Therapeutics, Inc. | Procédés de criblage de spécificité associée à un condensat et leurs utilisations |
EP4073251A1 (fr) * | 2019-12-13 | 2022-10-19 | Alnylam Pharmaceuticals, Inc. | Agents et compositions d'arni ciblant c9orf72 - cadre de lecture ouvert 72 sur le chromosome 9 - humain et procédés d'utilisation |
WO2021159008A2 (fr) | 2020-02-07 | 2021-08-12 | Maze Therapeutics, Inc. | Compositions et méthodes de traitement de maladies neurodégénératives |
US20250011773A1 (en) | 2021-04-06 | 2025-01-09 | Maze Therapeutics, Inc. | Compositions and methods for treating tdp-43 proteinopathy |
WO2023108071A2 (fr) * | 2021-12-08 | 2023-06-15 | Thomas Jefferson University | Méthodes relatives à la sclérose latérale amyotrophique |
WO2023220740A2 (fr) * | 2022-05-12 | 2023-11-16 | The Regents Of The University Of California | Approches d'édition de gène crispr/cas9 thérapeutique pour une mutation de type expansion de répétition c9orf72 en ipscs |
WO2024077109A1 (fr) | 2022-10-05 | 2024-04-11 | Maze Therapeutics, Inc. | Oligonucléotides antisens unc13a et leurs utilisations |
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WO2014062736A1 (fr) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Procédés permettant de surveiller l'expression de c9orf72 |
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Also Published As
Publication number | Publication date |
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EP2906696B1 (fr) | 2019-10-09 |
EP2906696A1 (fr) | 2015-08-19 |
US20150267197A1 (en) | 2015-09-24 |
DK2906696T4 (da) | 2023-02-27 |
ES2762326T5 (es) | 2023-04-27 |
DK2906696T3 (da) | 2019-11-25 |
EP2906696B2 (fr) | 2022-12-14 |
WO2014062686A1 (fr) | 2014-04-24 |
ES2762326T3 (es) | 2020-05-22 |
FI2906696T4 (fi) | 2023-03-18 |
US9963699B2 (en) | 2018-05-08 |
PT2906696T (pt) | 2019-12-16 |
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